TS-037741 —
T
he SMN2 gene contains sequences that regulate t
he level of inclusion of SMN exon7 in SMN mRNA, and some of t
hese act in a negative manner. Antisense o
ligonucleotides to block t
he negative regulating protein binding to t
hese sites can increase inclusion of SMN exon 7 and thus, t
he amount of SMN being produced.
Spinal muscular atrophy (SMA) is an inherited disease affecting the central nervous system, peripheral nervous system, and skeletal muscle. It is present primarily in infants and children but can also develop in adults. About 10,000 to 25,000 children and adults are living with the disease in the …